发明名称 GENERATION OF HIGH TITERS OF RECOMBINANT AAV VECTORS
摘要 <p><P>PROBLEM TO BE SOLVED: To provide a method for producing an adeno-associated virus (AAV) vector useful for human gene therapy in a sufficient amount. <P>SOLUTION: Disclosed is a method for generating high titers of recombinant AAV vectors which are available for delivery systems, wherein the method comprises a process for incubating cells under a condition enabling replication and packaging of AAV, wherein the cells comprises (a) a vector providing at least one intact copy of a stably integrated recombinant AAV vector in the cells, wherein the AAV vector comprises AAV inverted terminal repeat (ITR) regions and a transcription promoter operably linked to a target polynucleotide, and wherein the expression of the rep gene is limiting in the cells; and (b) a plasmid providing an AAV packaging plasmid that allows expression of the product of the rep gene, wherein in the plasmid the rep gene is operably linked to a heterologous promoter, and recombinant AAV vectors are generated thereby. <P>COPYRIGHT: (C)2005,JPO&NCIPI</p>
申请公布号 JP2005110694(A) 申请公布日期 2005.04.28
申请号 JP20050000789 申请日期 2005.01.05
申请人 TARGETED GENETICS CORP;JOHNS HOPKINS UNIV 发明人 FLOTTE TERENCE R;CARTER BARRIE J;GUGGINO WILLIAM B;SOLOW RIKKI
分类号 C12N15/09;A61K31/7088;A61K35/76;A61K38/00;A61K48/00;A61P1/18;A61P3/12;A61P7/06;A61P11/00;A61P43/00;C12N5/10;C12N7/00;C12N15/864;(IPC1-7):C12N15/09;A61K31/708 主分类号 C12N15/09
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